Success in the gene therapy space relies on embracing technological advances in upstream and downstream processes

HURDLES PREVENTING GENE THERAPIES FROM REACHING PATIENTS
Over the past two decades, gene therapies have seen continuous improvements in efficacy, safety and manufacturability, made possible by groundbreaking research in virology and advancements in manufacturing techniques and technologies. Understanding the potential these improvements bring, the U.S. FDA has stated that by 2025, it anticipates 10-20 new cell and gene therapy product approvals per year (1). This projection is based on the number of ongoing clinical trials, with 522 gene therapies currently at phases 1, 2 or 3 (2).

Although continued research holds the promise of enabling the development of increasingly powerful gene therapies, a prominent hurdle currently stands in the way of delivering these new medicines to the patients who need them. Production costs remain a fundamental concern for gene therapy developers. We are now seeing gene therapy drug prices in the multimillion-dollar range, with AAV-based gene therapies consistently taking the top spot for “the most expensive therapy in the world.” The title currently sits with Hemgenix, a one-off infusion treating hemophilia B, costing $3.5 million a dose (3).